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Results for “Inherited Bone Marrow Failure Syndromes”

208 trials

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Showing 20 of 208 results

Testing effectiveness (Phase 2)Study completedNCT04269889
What this trial is testing

Treatment of Refractory Diamond-Blackfan Anemia With Eltrombopag

Who this might be right for
Anemia, Diamond-Blackfan
National Heart, Lung, and Blood Institute (NHLBI) 15
Not applicableEnded earlyNCT04275479
What this trial is testing

Diabetes/ Endocrine Surveillance in SDS

Who this might be right for
Shwachman-Diamond Syndrome
Washington University School of Medicine 11
Not applicableStudy completedNCT01114776
What this trial is testing

Multi-Center Study of Iron Overload: Pilot Study

Who this might be right for
Sickle Cell DiseaseThalassemiaDiamond-Blackfan Anemia
UCSF Benioff Children's Hospital Oakland 20
Early research (Phase 1)Study completedNCT00569283
What this trial is testing

Donor Natural Killer Cell Infusion in Preventing Relapse or Graft Failure in Patients Who Have Undergone Donor Bone Marrow Transplant

Who this might be right for
Cancer
Asan Medical Center 18
Testing effectiveness (Phase 2)Active Not RecruitingNCT03924401
What this trial is testing

Acute GVHD Suppression Using Costimulation Blockade to Expand Non-malignant Transplant

Who this might be right for
Graft Versus Host Disease
Emory University 30
Testing effectiveness (Phase 2)Study completedNCT00229619
What this trial is testing

Rituximab to Treat Moderate Aplastic Anemia, Pure Red Cell Aplasia, or Diamond Blackfan Anemia

Who this might be right for
Anemia, AplasticRed-Cell Aplasia, PureAnemia, Diamond-Blackfan
National Heart, Lung, and Blood Institute (NHLBI) 11
Large-scale testing (Phase 3)Study completedNCT00235391
What this trial is testing

Expanded Access of Deferasirox to Patients With Congenital Disorders of Red Blood Cells and Chronic Iron Overload

Who this might be right for
ThalassemiaSickle Cell DiseaseDiamond Blackfan Anemia+1 more
Novartis Pharmaceuticals 1,683
Early research (Phase 1)Study completedNCT01720147
What this trial is testing

Quercetin in Children With Fanconi Anemia; a Pilot Study

Who this might be right for
Fanconi Anemia
Children's Hospital Medical Center, Cincinnati 30
Testing effectiveness (Phase 2)Active Not RecruitingNCT01659606
What this trial is testing

Radiation- and Alkylator-free Bone Marrow Transplantation Regimen for Patients With Dyskeratosis Congenita

Who this might be right for
Dyskeratosis CongenitaHoyeraal Hreidarsson SyndromeRevesz Syndrome+1 more
Boston Children's Hospital 40
Not applicableLooking for participantsNCT07145411
What this trial is testing

HPC Offered for PRESERVE Expansion

Who this might be right for
Bone Marrow Transplant (BMT)
Ossium Health, Inc. 10
Not applicableLooking for participantsNCT05687149
What this trial is testing

Defining the Natural History of Squamous Cell Carcinoma in Fanconi Anemia

Who this might be right for
Fanconi AnemiaInherited Bone Marrow Failure Syndrome
National Cancer Institute (NCI) 200
Testing effectiveness (Phase 2)Study completedNCT01044186
What this trial is testing

A Protocol to Allow Treatment With ICL670 for Patients With or at Risk of Life-threatening Complications of Transfusional Iron Overload Who Are Unable to Tolerate Other Iron Chelators Because of Documented Severe Toxicity

Who this might be right for
Transfusional Iron Overload
Novartis Pharmaceuticals 30
Not applicableStudy completedNCT00499070
What this trial is testing

Assessing Immune Function in Young Patients With Cytopenia That Did Not Respond to Treatment

Who this might be right for
Dyskeratosis CongenitaFanconi AnemiaMyelodysplastic Syndromes+2 more
University Hospital Freiburg 119
Testing effectiveness (Phase 2)WithdrawnNCT00816413
What this trial is testing

Donor Stem Cell Transplant, Pentostatin, and Total-Body Irradiation in Treating Patients With Hematological Cancer

Who this might be right for
Chronic Myeloproliferative DisordersLeukemiaLymphoma+2 more
University of Nebraska
Not applicableStudy completedNCT00271089
What this trial is testing

Blood Cell Collection for Future Use in Individuals With Fanconi Anemia

Who this might be right for
Fanconi Anemia
National Heart, Lung, and Blood Institute (NHLBI) 40
Not applicableActive Not RecruitingNCT05236764
What this trial is testing

Haploidentical Hematopoietic Cell Transplantation Using TCR Alpha/Beta and CD19 Depletion

Who this might be right for
Acute Lymphoblastic Leukemia in RemissionAcute Myeloid Leukemia in RemissionMyelodysplastic Syndromes+8 more
Baylor College of Medicine 3
Testing effectiveness (Phase 2)Ended earlyNCT00617929
What this trial is testing

Antithymocyte Globulin, Clofarabine, and Rituximab in Treating Patients After an Unsuccessful Stem Cell Transplant

Who this might be right for
Cancer
Masonic Cancer Center, University of Minnesota 12
Testing effectiveness (Phase 2)Looking for participantsNCT03653338
What this trial is testing

T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other Anemias

Who this might be right for
Sickle Cell AnemiaBeta-thalassemia MajorDiamond-blackfan Anemia
Paul Szabolcs 5
Testing effectiveness (Phase 2)Looking for participantsNCT06839456
What this trial is testing

Phase 1/2: CD45RA Depleted Stem Cell Addback to Prevent Viral or Fungal Infections Post TCRab/CD19 Depleted HSCT

Who this might be right for
LeukemiaHigh Risk Acute Lymphoblastic LeukemiaHigh Risk Acute Myeloid Leukemia+11 more
Children's Hospital of Philadelphia 100
Testing effectiveness (Phase 2)Looking for participantsNCT03128996
What this trial is testing

Reduced Intensity Conditioning and Familial HLA-Mismatched BMT for Non-Malignant Disorders

Who this might be right for
Severe Sickle Cell DiseaseBone Marrow Failure SyndromesMetabolic Disorders+3 more
Washington University School of Medicine 29
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